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Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Global Blood Therapeutics, Inc., a biopharmaceutical company, engages in the discovery, development, and delivery of treatments for underserved patient communities with sickle cell disease (SCD). The company offers Oxbryta tablets, an oral, once-daily therapy for SCD. It also evaluates the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase II a clinical trial of adolescent and pediatric patients with SCD. In addition, the company is developing its lead product candidate inclacumab, novel human monoclonal antibody, which is in Phase III a clinical trial to reduce the incidence of painful vaso-occlusive crises, and resulting hospital admissions; and GBT601, a hemoglobin polymerization inhibitor that is in Phase I development. It has a license and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD and beta thalassemia; and Sanofi S.A. to two early-stage research programs in SCD. Global Blood Therapeutics, Inc. was incorporated in 2011 and is headquartered in South San Francisco, California.

Hypera S.A. operates as a pharmaceutical company in Brazil. It offers prescription products under the Adacne, Addera, apri, AmpliumG, please, Celestamine, Celestone, Celestone Soluspan, Cizax, deciprax, Derive C Micro, Micro Drift, Dermotil Fusid, Digedrat, diprogent, Diprosalic, Diprosone, diprospan, Emprol XR, Flow, Garasone, Halobex, Lipanon, moon, Lydian, macrodantin, Max Sulid, milgamma, Mioflex A, nesina, Novotram, oximax, peridal, Peridal Suspension, PredSim, Pressaliv, Quadriderm, Rizi, Rizi M, softalm, tacroz, tinodin, umma, and velunid brands. The company also provides sun protection and moisturizing skin care products under the Mantecorp Skincare brands; similar products under the Doralgina, Dropy D, balance, Flavonid, histamin, NeoFresh, neolefrin, Neochemistry Vitamins, Neosorum, and Torsilax brand names; and consumer health products under the Apracur, Benegrip, Buscopan, Coristina D Pro, Engov, Epocler, Estomazil, and Neosaldina brands, as well as similar and generic drugs under the Neo Química brand. In addition, it offers nutritional and vitamin supplement products under the Tamarine, Vitasay, Biotônico Fontoura, and Zero-Cal brands; and generic medicines under the Hydroxyzine, Sodium Diclofenac, Dipyron, Ibuprofen, Losartan Potassium, Mal Dexchlorpheniramine, Naproxene, Paracetamol, Simethicon, Loratadine, Omeprazole, Tadalaphyl, and Desogestrel brand names. The company was formerly known as Hypermarcas S.A. and changed its name to Hypera S.A. in February 2018. Hypera S.A. was founded in 2001 and is headquartered in São Paulo, Brazil.

Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product is ARIKAYCE® (amikacin liposome inhalation suspension), which is approved in the United States for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a rare and often chronic infection that can cause irreversible lung damage and can be fatal. Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009, an inhaled formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.

Principia Biopharma Inc., a late-stage biopharmaceutical company, focuses on developing novel therapies for immunology and oncology. The company is developing PRN1008, an inhibitor that is in Phase III clinical trial for the treatment of pemphigus, a chronic skin disease, as well as in Phase II clinical trial to treat immune thrombocytopenic purpura. It is also developing PRN2246/SAR442168, an inhibitor, which has completed Phase I clinical trial for treating multiple sclerosis (MS) and other central nervous system (CNS) diseases; PRN1371, a drug candidate that is in Phase I clinical trial for the treatment of solid tumors; and oral small molecule inhibitors of the immunoproteasome. The company has a collaboration agreement with Genzyme Corporation to develop relapsing and progressive MS and other diseases of the CNS. Principia Biopharma Inc. was incorporated in 2008 and is headquartered in South San Francisco, California.